Even under the best of circumstances, the CRISPR gene-editing technology that we explore in this issue of OncologyLive
won’t make it into clinical practice any time soon. But developments are moving so quickly in the oncology field that it is important to keep abreast of what certainly seems to be an important new tool for drug discovery.
Imagine, for example, being able to knock out the gene that encodes PD-1, the negative immune system regulator whose activity is targeted in the emerging class of checkpoint blockade monoclonal antibodies. Instead of using an antibody to attack PD-1, a drug that would delete the gene could be used instead.
That is shaping up as the first mission of US and Chinese researchers seeking to launch clinical trials employing the gene-editing technique, as our correspondent reports in our cover story, “Why CRISPR Gene-Editing Technology Is Captivating Research Field.”
Of course, there are many questions to be answered about the new technology. It is not clear how much risk patients would face if they volunteered to join a CRISPR clinical trial. The potential for off-target effects are particularly troublesome; one scientist contends the algorithms researchers are using to predict those effects are not accurate enough to capture what would happen if these techniques are employed.
Certainly, these questions must be answered before any therapy developed through CRISPR is tried on a patient. As panel members made clear during a National Institutes of Health committee hearing in June, individuals should have a clear idea of the risks they face before consenting to participate in a CRISPR clinical trial.
We can understand this trepidation.
Although we cannot be paralyzed by the potential risks of any new therapy, the precarious nature of gene therapy calls for an abundance of caution. We also will have to maintain perspective as the capabilities of this new technology unfold in preclinical and clinical experiments. As with other potential anticancer advances, we’ve already seen many superlative descriptions of this new technology.
Yet much work must be done not only to prove the technology is safe but also to show that it is more effective than currently available methods—or at least effective enough to make the risks worthwhile.